Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT03164135)

Disease info:

HIV stands for human immunodeficiency virus. It harms the immune system by destroying the white blood cells that fight infection. This puts at risk for serious infections and certain cancers.




Worldwide, there were about 1.7 million new cases of HIV in 2018. About 37.9 million people were living with HIV around the world in 2018, and 23.3 million of them were receiving medicines to treat HIV, called antiretroviral therapy (ART).
Official title:
Safety and Feasibility Study of Allotransplantation of CRISPR/Cas9 CCR5 Gene Modified CD34+ Hematopoietic Stem/Progenitor Cells in HIV-infected Subjects With Hematological Malignances

Principal Investigator: Hu Chen, MD, PhD  Affiliated Hospital of Academy to Military Medical Sciences

Principal Investigator: Hongkui Deng, PhD  Affiliated Hospital of Academy to Military Medical Sciences       

Principal Investigator: Hao Wu, MD, PhD  Affiliated Hospital of Academy to Military Medical Sciences


Affiliated Hospital to Academy of Military Medical Sciences


China, Beijing

Study start:
May. 23, 2017
5 participants
Gene editing method:
Type of edit:
Gene ablation
CCR5 C-C motif chemokine receptor 5
Delivery method:
non-viral - Ex-vivo
Phase not applicable
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


The investigators performed this study to evaluate the safety and feasibility of transplantation with CRISPR-Cas9 CCR5 gene modified CD34+ hematopoietic stem/progenitor cells for patients that develop AIDS and hematological malignances. Patients will be treated with antiviral therapy (ART) to achieve undetectable HIV-1 virus in peripheral blood before conditioning. CD34+ cells from donors will be infused into the patients after treatment with CRISPR-Cas9 to ablate CCR5 gene.

Last updated: Apr. 10, 2022
Source: US National Institutes of Health (NIH)
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